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Clinical Trials

This information is provided by ClinicalTrials.gov

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Displaying 1-10 of 14 results.
Lentiviral Gene Therapy for X-ALD
Status: Recruiting
Last Changed: Sep 19, 2019
First Received: Nov 01, 2018
Disease(s): X-linked Adrenoleukodystrophy
Intervention(s): Intracerebral LV gene therapy
Locations: Shenzhen Geno-immune Medical Institute, Shenzhen, Guangdong, China
Minnesota Adrenoleukodystrophy Registry Study (MARS) and Biobank
Status: Recruiting
Last Changed: Jun 06, 2019
First Received: Dec 31, 2018
Disease(s): ALD (Adrenoleukodystrophy)
Intervention(s): Medical Record Abstraction, Biospecimen Sample Collection
Locations: Masonic Cancer Center at University of Minnesota, Minneapolis, Minnesota, United States
Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
Status: Recruiting
Last Changed: Feb 18, 2020
First Received: Feb 25, 2019
Disease(s): Cerebral Adrenoleukodystrophy (CALD)
Intervention(s): Genetic
Locations: Lucile Packard Children's Hospital, Palo Alto, California, United States
Boston Children's Hospital/Massachusetts General Hospital, Boston, Massachusetts, United States
University of Minnesota, Minneapolis, Minnesota, United States
Hôpital Robert Debré, Paris, France
Ospedale Pediatrico Bambino Gesù, Rome, Italy
... and 2 other locations.
A Study to Prospectively Assess Disease Progression in Male Children With X-ALD
Status: Recruiting
Last Changed: Aug 08, 2018
First Received: Sep 12, 2017
Disease(s): X-Linked Adrenoleukodystrophy
Locations: Stanford University, Palo Alto, California, United States
Massachusetts General Hospital, Boston, Massachusetts, United States
University of Pittsburgh, Pittsburgh, Pennsylvania, United States
Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy
Status: Recruiting
Last Changed: Sep 24, 2015
First Received: Sep 24, 2015
Disease(s): Metachromatic Leukodystrophy, Adrenoleukodystrophy
Intervention(s): transduced CD34+ hematopoietic stem cell
Locations: Shenzhen Second People's Hospital, The First Affiliated Hospital of Shenzhen University, Shenzhen, Guangdong, China
Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children
Status: Not yet recruiting
Last Changed: Feb 11, 2020
First Received: Aug 28, 2018
Disease(s): Xlsma, DMD, X-ALD, TSC
Locations: Children's Hospital of Fudan University, Shanghai, China
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
Status: Recruiting
Last Changed: Mar 22, 2019
First Received: Dec 08, 2017
Disease(s): Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy, Inherited Metabolic Disorders
Intervention(s): Blood and Marrow Transplant
Locations: Masonic Caner Center at University of Minnesota, Minneapolis, Minnesota, United States
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Status: Recruiting
Last Changed: Dec 03, 2019
First Received: Oct 02, 2014
Disease(s): Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses
Intervention(s): DUOC-01
Locations: Duke University Medical Center, Durham, North Carolina, United States
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Status: Recruiting
Last Changed: Sep 26, 2019
First Received: Jun 23, 2014
Disease(s): Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
Intervention(s): Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
Locations: Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
Status: Recruiting
Last Changed: Jan 28, 2020
First Received: May 01, 2018
Disease(s): Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy
Intervention(s): Thiotepa--single daily dose, Thiotepa--escalated dose
Locations: UF Health Shands Children's Hospital, Gainesville, Florida, United States